A Promoter Specifically Active in Newly Forming and Remodeling Arteries

Case ID:

2008-024-a

Web Published:

11/7/2012

Description:

Current State of the Art:

Studies in angiogenesis link new blood vessel formation to both
wound healing and pathologies in the human body. Angiogenesis can be stimulated
during regeneration of diseased or damaged tissues as well as during tumor
growth and metastasis, in order to gain access to oxygen and nutrients necessary
for survival. Gene therapy is a promising field to combat human disease by
integrating new healthy gene products into a diseased or damaged state to
influence recovery or impede disease progression.

The Global gene therapy market is still in the experimental stages
with only a few products in Phase II/III clinical trials. One gene therapy drug,
Gendicine, has been approved and is marketed in China. Overall the industry is
predicted to reach $480 million by 2015.

Disadvantages
with the Current Art:

Companies moving to develop gene therapy technologies would benefit
from a built-in modifier or a controlled, targeted aspect of the delivery of the
therapeutic gene product. Otherwise irrevocable genetic changes made to the
patient’s cells can have detrimental effects.

Advantages
of Invention:

The inventors have engineered an optimized Alk1 promoter fragment
which can be used in a variety of constructs including but not limited to an
adenoviral vector. Endothelial cells containing therapeutic genes with this
promoter will only express the therapeutic product in newly formed or remodeling
arteries.

The inventors have developed a mouse model system that can be used
to identify drugs that would be useful in either inhibiting or promoting new
blood vessel formation.